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Blog Category
Research and Critical Trials
Total posts in Category: 29
HNF Awards the inaugural HNF Clinical Translation Fellowship
HNF Awards the inaugural HNF Clinical Translation Fellowship in the amount of $170,000 to Kayla...
Breaking News for SORD Deficiency Patients
The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.
HNF Contributes CMTRN Data to C-Path’s RDCA-DAP
HNF Contributes CMTRN Data to C-Path’s RDCA-DAP, Strengthening Research Capabilities for Inherited...
Breaking News: HNF to Host “Revolutionizing Clinical Trials” Panel at Bio 2024
HNF is thrilled to announce that we have been accepted to host a 1-hour panel discussion during the Bio International Convention in San Diego, CA, in June 2024.
CMT Biobank: We Need Your HELP!
HNF is excited to continue collecting samples for researchers and industry to help develop treatments for CMT.
Reducing Genetic Testing Barriers for the Charcot-Marie-Tooth Community
Hereditary Neuropathy Foundation Partners with InformedDNA® on CMT Genie Project
Novartis CMT1A Update
In July 2023, Novartis announced the acquisition of DTx Pharma, a San-Diego based, preclinical stage biotechnology company focused on leveraging its proprietary FALCON platform to develop siRNA therapies for neuroscience indications.
Congratulations to GRIN Advisory Board member, Professor Joshua Burns
Congratulations to GRIN Advisory Board member, Professor Joshua Burns, for being recognized by the Australian Academy of Health and Medical Sciences for his outstanding contributions to medical and health sciences.
Pediatric CMT Natural History Study Enrollment Opening at Two Centers of Excellence
Pediatric CMT Natural History Study enhancement to their Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN) patient registry, Global Registry for Inherited Neuropathies (GRIN).
DTx Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Dtx today announced that the FDA has granted Orphan Drug Designation to DTx-1252, an investigational FALCON small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Great News From HNF TRIAD Industry Partner — Pharnext
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003
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Faces of CMT – Bernadette (9)
Faces of CMT – CMT4A (7)
Faces of CMT – Grace (18)
Faces of CMT – HELP Fund (8)
Faces of CMT – HNPP Fund (3)
Faces of CMT – James (1)
Faces of CMT – Jaxson (7)
Faces of CMT – Zach (18)
Featured (28)
GRIN Patient Registry (39)
HNF Team (10)
Industry (3)
Living with CMT (78)
Medical professionals (2)
Movement Is Medicine (17)
Optic Hereditary Neuropathy (5)
Our Impact (3)
Clinical Trials (20)
PFDD (1)
PFDD Meeting 28SEP2018 (5)
Past Events (35)
Patience Resources (0)
Accessible Travel (4)
Bracing (11)
Breathing (3)
Canine Companions (5)
Cannabis (8)
Caregivers (3)
Dating and Relationships (7)
Emotional and Mental Health (9)
Family Planning (1)
Fashion and Products (1)
Genetic Testing (11)
Home Accessibility (2)
Inclusive Employment (3)
Inspire Community (7)
Legal Rights and Benefits (8)
Neurotoxic Drugs (4)
Newly Diagnosed (7)
Nutrition (3)
Occupational Thearpy (2)
Pain (8)
Podiatry (4)
PT and Exercise (25)
Research and Critical Trials (29)
School & College (6)
Surgery (10)
Telemedicine (1)
Tips and Hacks (2)
Patient-Focused Research (1)
Pediatrics & CMT (10)
Registry (16)
Research (95)
School Outreach Program and Team CMT Kids (1)
Special Events (2)
Bike New York (19)
Other Events (4)
Spin-a-thon (7)
TCS New York City Marathon (7)
Team CMT Members (61)
The CMT Genie (6)
TRIAD (27)
TRIAD – Clinical Trial Readiness (5)
Vitaccess (1)
TRIAD – Research Gifts (0)
University of Helsinki (2)
Veneto (1)
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Burke Insitute (3)
University of Cambridge (1)
University of Miami (1)
University of San Antonio Texas (1)
TRIAD – Therapeutics (4)
Applied Therapeutics (10)
Pharnext (10)
Rarebase (4)
Types of CMT (1)
CMT Type – CMT1A (21)
CMT Type – CMT1B (1)
CMT Type – CMT1X (2)
CMT Type – CMT2A (8)
CMT Type – CMT2C (4)
CMT Type – CMT4A GDAP1 (3)
CMT Type – CNTNAP1 (1)
CMT Type – GAN (5)
CMT Type – HDAC6 (1)
CMT Type – HNPP (3)
CMT Type – MTRFR-C12orf65 (6)
CMT Type – SORD Deficiency (9)
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